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Journal article

Accumulation of glycosphingolipids in Niemann-Pick C disease disrupts endosomal transport.

Abstract:

Glycosphingolipids are endocytosed and targeted to the Golgi apparatus but are mistargeted to lysosomes in sphingolipid storage disorders. Substrate reduction therapy utilizes imino sugars to inhibit glucosylceramide synthase and potentially abrogate the effects of storage. Niemann-Pick type C (NPC) disease is a disorder of intracellular transport where glycosphingolipids (GSLs) and cholesterol accumulate in endosomal compartments. The mechanisms of altered intracellular trafficking are not k...

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Publication status:
Published

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Publisher copy:
10.1074/jbc.m311591200

Authors


te Vruchte, D More by this author
Lloyd-Evans, E More by this author
Veldman, RJ More by this author
Neville, DC More by this author
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Journal:
The Journal of biological chemistry
Volume:
279
Issue:
25
Pages:
26167-26175
Publication date:
2004-06-05
DOI:
EISSN:
1083-351X
ISSN:
0021-9258
URN:
uuid:9dd1976c-fd0b-4e2b-b1c8-518035bf0290
Source identifiers:
185150
Local pid:
pubs:185150

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